In Vivo Therapeutic Platform Based on RNA Base Editing
The RNA base editing platform aims to develop therapies for Hurler syndrome, the most severe subtype of mucopolysaccharidosis type I which include diseases related to liver, muscles and nerve system, etc. based on the RNA single-base editing technology LEAPER™.
LEAPER™’s purpose is to edit RNA programmatically through endogenous ADAR (adenosine deaminase acting on RNA, i.e. the RNA adenosine deaminase).
Significant number of single-gene genetic diseases caused by single point mutation, of which a significant portion is G>A; Current ERT’s cost is high and with high ADA risk
Using an RNA molecule of <100nt to edit the mutant RNA, specifically converting significant portion of G>A mutation back to I/G, resulting in expression of wild type, endogenous protein
Papers & Presentations
- In July 2019, LEAPER™ was first published in Nature Biotechnology; In February 2022, an upgraded version of LEAPER™ (LEAPER™ 2.0) that shows significantly improved efficiency and fidelity of RNA editing in vitro and in vivo was published in Nature Biotechnology;
- In May 2020, EdiGene presented data on its lead therapeutic program for patients with the most severe form of mucopolysaccharidosis type I (MPS I), known as Hurler syndrome, at the 23rd American Society of Gene & Cell Therapy (ASGCT) Annual Meeting